How can we give an experimental drug to a human being for the first time…safely ??

Every medication prescribed or otherwise can cause side effects. By the time a medication is on a shelf somewhere, it has been extensively tested in an appropriate number of people and a list of risks, side effects, contraindications, drug interactions, study findings and post marketing findings are available.

Giving an experimental drug to a human for the first time is quite different. By definition, we have no knowledge of how the drug will behave in a human being including basic side effects, blood or organ toxicities, drug interactions and serious adverse reactions.

There are a number of ways safety can be maximised in this situation.

Develop a new drug as similar to an existing drug as possible by making small changes that offer benefit to the new drug with the hope that the small change doesn’t affect drug behaviour

Have multiple test drugs in development so that the best behaving drug can be chosen for further development

Use known and safe methods of drug development and chemical or biological manufacturing

Follow international standards and guidelines for drug development and animal testing

Test the drug in a range of animal models at a range of doses and for a range of treatment periods

Look for drug efficacy in disease animal models and look for drug toxicity and metabolism in appropriate animal models able to detect certain toxicities

Obtain only high quality laboratory and animal data

Plan a step wise drug exposure study in which groups of volunteers are progressively exposed to higher doses of the drug after starting at an extremely low level compared to levels given to the animals in drug development

Choose healthy volunteers after extensive examination and testing for any factors that could put a volunteer at risk

Give the drug in a specialised facility with experience at giving experimental drugs and with experienced senior Principal Investigators

Have regular and dynamic real time review of participants in order to detect toxicities early and manage appropriately or hold the dosing or study until resolved

Always maintain standards and guidelines in accordance with Good Clinical Practice

Maintain high quality recording of data and invite regulatory agencies and monitors to review all data and processes regularly to maintain standards and safety